Month: October 2020

We are pleased to share with you that on October 22, 2020, The US Food and Drug Administration (FDA) approved the first drug to treat COVID-19: Veklury (remdesivir), an antiviral medicine manufactured by Gilead Sciences Inc. to be administered  IV to patients needing hospitalization. Veklury-Remdesivir was originally developed to treat the Ebola virus, however, did not meet the efficacy endpoint in the Ebola clinical trial. In a large study led by the US National Institutes of Health (NIH), it was shown that the drug shortened the course of illness from an average of 15 days to about 10 days in hospitalized patients.

Veklury was submitted under the New Drug Application (NDA) track and granted a Fast Track designation by the FDA, which among other things, maximized the opportunities for Gilead to engage with the agency in its development of Veklury, for the treatment of COVID-19. Based on this designation, on April 6th, 2020, FDA granted Gilead’s request and accepted its proposal to allow for a rolling review of its development program for Veklury. Under this process, Gilead could submit the NDA sections for FDA review as they appear. Under traditional processes, the FDA’s review of an NDA does not begin until the sponsor has submitted the entire application to the Agency.

Notably, on the basis of the Department of Health and Human Services (HHS) determination that there is a public health emergency that involves the virus that causes COVID-19, and while Veklury was an investigational drug and not approved for any indication, the FDA issued (on February 2020) an Emergency Use Authorization (EUA) for emergency use of Veklury for the treatment of suspected or laboratory-confirmed COVID-19 patients. Emergency use authorization is NOT the same as FDA approval or licensure. EUAs do not remain in effect indefinitely and FDA is continuously evaluating the continued appropriateness of the EUA.

In summary, from the approval process of Veklury, we can learn that nowadays the FDA is committed to expediting the development and availability of COVID-19 treatments. As part of the FDA’s Coronavirus Treatment Acceleration Program, the agency is focusing on moving new medical products to patients as soon as possible, while at the same time determining whether they are effective and if their benefits outweigh their risks.

This Newsletter Prepared by:

Tsufit Gross, Ph.D

Pharma and Biotechnology project manager

For more information about our services visit:

The Nobel Prize for Chemistry was awarded this year for the invention of Genetic Scissors: a tool for rewriting the code of life.

Emmanuelle Charpentier and Jennifer A. Doudna have discovered one of gene technology’s sharpest tools: the CRISPR/Cas9 genetic scissors. Using these, the DNA of animals, plants and microorganisms can be changed/edited with extremely high precision.

This technology has had a revolutionary impact on the life sciences, is contributing to new cancer therapies and may make the dream of curing inherited diseases come true.

Since the discovery of CRISPR/Cas9, the research of this tool has led to a blooming landscape of pre-clinical and clinical studies in humans.
FDA considers any use of CRISPR/Cas9 gene editing in humans to be gene therapy, thus requiring extensive regulatory efforts in order to bring such products from early concept to clinical application. 

Gene therapy products are regulated by the FDA’s Center for Biologics Evaluation and Research (CBER). Clinical studies of gene therapy in humans require the submission of an investigational new drug application (IND) prior to their initiation in the United States, and marketing of a gene therapy product requires submission and approval of a biologics license application (BLA). 

At Gsap, our team of Advanced Therapies experts is excited to be at the frontier of this field, with a unique portfolio of process development, preclinical, clinical and regulatory services, assisting our clients to bring gene-therapy products from early POC to realization into clinical use.

If you develop a gene-editing product, do not hesitate to contact us!

This Newsletter Prepared by:

Diana Gershtein, M.Sc., M.B.A.

Cell Therapy Section Manager

For more information about our services visit: